Gene, RNA, and ASO-based Therapeutic Approaches in Cystic Fibrosis

Overview

Journal J Cyst Fibros

Publisher Elsevier

Specialty Pulmonary Medicine

Date 2023 Jan 19

PMID 36658041

Authors

Normand E Allaire

Uta Griesenbach

Batsheva Kerem

John D Lueck

Noemie Stanleigh

Yifat S Oren

Affiliations

Soon will be listed here.

Abstract

Most people with Cystic Fibrosis (PwCF) harbor Cystic Fibrosis Transmembrane Conductance (CFTR) mutations that respond to highly effective CFTR modulators (HEM); however, a small fraction of non-responsive variants will require alternative approaches for treatment. Furthermore, the long-term goal to develop a cure for CF will require novel therapeutic strategies. Nucleic acid-based approaches offer the potential to address all CF-causing mutations and possibly a cure for all PwCF. In this minireview, we discuss current knowledge, recent progress, and critical questions surrounding the topic of Gene-, RNA-, and ASO-based therapies for the treatment of Cystic Fibrosis (CF).

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